EP2475376A4 - FORMULATIONS FOR LYSOSOMAL ENZYMES - Google Patents

FORMULATIONS FOR LYSOSOMAL ENZYMES

Info

Publication number
EP2475376A4
EP2475376A4 EP10790224.9A EP10790224A EP2475376A4 EP 2475376 A4 EP2475376 A4 EP 2475376A4 EP 10790224 A EP10790224 A EP 10790224A EP 2475376 A4 EP2475376 A4 EP 2475376A4
Authority
EP
European Patent Office
Prior art keywords
formulations
lysosomal enzymes
lysosomal
enzymes
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
EP10790224.9A
Other languages
German (de)
French (fr)
Other versions
EP2475376A2 (en
EP2475376B1 (en
Inventor
Jonathan H Lebowitz
Byeong Chang
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Biomarin Pharmaceutical Inc
Original Assignee
Biomarin Pharmaceutical Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Biomarin Pharmaceutical Inc filed Critical Biomarin Pharmaceutical Inc
Priority to EP16153694.1A priority Critical patent/EP3075386B1/en
Priority to EP19203438.7A priority patent/EP3679942A1/en
Publication of EP2475376A2 publication Critical patent/EP2475376A2/en
Publication of EP2475376A4 publication Critical patent/EP2475376A4/en
Application granted granted Critical
Publication of EP2475376B1 publication Critical patent/EP2475376B1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22Hormones
    • A61K38/30Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Dermatology (AREA)
  • Endocrinology (AREA)
  • Neurology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
EP10790224.9A 2009-06-17 2010-06-17 Formulations for lysosomal enzymes Active EP2475376B1 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
EP16153694.1A EP3075386B1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes
EP19203438.7A EP3679942A1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US18774709P 2009-06-17 2009-06-17
PCT/US2010/039083 WO2010148253A2 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes

Related Child Applications (3)

Application Number Title Priority Date Filing Date
EP19203438.7A Division EP3679942A1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes
EP16153694.1A Division EP3075386B1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes
EP16153694.1A Division-Into EP3075386B1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes

Publications (3)

Publication Number Publication Date
EP2475376A2 EP2475376A2 (en) 2012-07-18
EP2475376A4 true EP2475376A4 (en) 2013-05-22
EP2475376B1 EP2475376B1 (en) 2016-03-30

Family

ID=43357058

Family Applications (3)

Application Number Title Priority Date Filing Date
EP19203438.7A Pending EP3679942A1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes
EP16153694.1A Active EP3075386B1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes
EP10790224.9A Active EP2475376B1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes

Family Applications Before (2)

Application Number Title Priority Date Filing Date
EP19203438.7A Pending EP3679942A1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes
EP16153694.1A Active EP3075386B1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes

Country Status (4)

Country Link
US (1) US8785168B2 (en)
EP (3) EP3679942A1 (en)
ES (2) ES2569514T3 (en)
WO (1) WO2010148253A2 (en)

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3782655A1 (en) 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
JP5627571B2 (en) 2008-05-07 2014-11-19 バイオマリン ファーマシューティカル インコーポレイテッド Lysosomal targeting peptides and uses thereof
US8540985B2 (en) 2008-06-26 2013-09-24 Orphazyme Aps Use of Hsp70 as a regulator of enzymatic activity
RU2013125923A (en) 2010-11-30 2015-01-10 Орфазиме Апс METHODS FOR INCREASING THE EXTRACELLULAR ACTIVITY OF HSP70
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
KR20230066482A (en) * 2012-03-07 2023-05-15 아미쿠스 세라퓨틱스, 인코포레이티드 High concentration alpha-glucosidase compositions for the treatment of pompe disease
ES2729997T3 (en) * 2012-11-27 2019-11-07 Biomarin Pharm Inc Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
AU2014218854B2 (en) 2013-02-20 2019-01-24 Valerion Therapeutics, Llc Methods and compositions for treatment of Pompe disease
CN105431527A (en) * 2013-06-11 2016-03-23 比奥卡尔齐斯股份有限公司 Biomolecule drying process for long-term storage
US20160346363A1 (en) * 2013-07-15 2016-12-01 New York University Methods and oral formulations for enzyme replacement therapy of human lysosomal and metabolic diseases
IL312865B2 (en) 2013-09-11 2025-06-01 Eagle Biologics Inc Liquid protein formulations containing viscosity-reducing agents
KR102058864B1 (en) 2013-12-16 2019-12-24 주식회사 티움바이오 Pharmaceutical composition having improved stability comprising a fusion protein of factor vii
US10709700B2 (en) 2014-09-15 2020-07-14 Orphazyme A/S Arimoclomol formulation
CN114540327B (en) 2014-09-30 2026-03-24 阿米库斯治疗学公司 Highly acidic α-glucosidase with enhanced carbohydrate activity
AU2015325055B2 (en) 2014-10-01 2021-02-25 Eagle Biologics, Inc. Polysaccharide and nucleic acid formulations containing viscosity-lowering agents
WO2016065319A1 (en) * 2014-10-24 2016-04-28 Shire Human Genetic Therapies, Inc. Lysosomal targeting of enzymes, and uses thereof
MA41022A (en) * 2014-11-24 2017-10-03 Shire Human Genetic Therapies LYSOSOMIAL TARGETING AND CORRESPONDING USES
US9981021B1 (en) 2015-04-09 2018-05-29 Kinetiq, Inc. Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof
JP5954916B1 (en) 2015-04-14 2016-07-20 中外製薬株式会社 A pharmaceutical composition for preventing and / or treating atopic dermatitis, comprising an IL-31 antagonist as an active ingredient
AU2016283344B2 (en) 2015-06-24 2022-08-04 Jcr Pharmaceuticals Co., Ltd. Fusion protein containing BDNF
KR20250007693A (en) 2015-12-30 2025-01-14 아미쿠스 세라퓨틱스, 인코포레이티드 Augmented acid alpha-glucosidase for the treatment of pompe disease
MX2018010196A (en) * 2016-02-24 2018-11-19 Biomarin Pharm Inc Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof.
AU2017239641A1 (en) * 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
SMT202200279T1 (en) * 2016-03-30 2022-09-14 Amicus Therapeutics Inc Formulations comprising recombinant acid alpha-glucosidase
EP3442530A1 (en) 2016-04-13 2019-02-20 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
EP3445451B1 (en) * 2016-04-21 2021-08-18 Baylor College of Medicine Compositions and methods for the treatment of lysosomal storage disorders and disorders characterized by lysosomal dysfunction
KR20210059796A (en) 2016-04-29 2021-05-25 오르파짐 에이/에스 Arimoclomol for treating glucocerebrosidase associated disorders
KR102497564B1 (en) * 2016-12-26 2023-02-07 제이씨알 파마 가부시키가이샤 Novel anti-human transferrin receptor antibody capable of penetrating blood-brain barrier
CA3052936A1 (en) 2016-12-26 2018-07-05 Jcr Pharmaceuticals Co., Ltd. Fusion protein of bdnf and anti-transferrin receptor antibody
SMT202300187T1 (en) 2017-05-15 2023-07-20 Amicus Therapeutics Inc Recombinant human acid alpha-glucosidase
DK3635009T3 (en) 2017-06-07 2026-03-30 Regeneron Pharma Compositions and methods for internalizing enzymes
US12258597B2 (en) 2018-02-07 2025-03-25 Regeneron Pharmaceuticals, Inc. Methods and compositions for therapeutic protein delivery
BR112020023145A2 (en) 2018-05-17 2021-02-02 Regeneron Pharmaceuticals, Inc. anti-cd63 antibody or antigen-binding fragment thereof, bispecific antigen-binding molecule, therapeutic protein of multiple domains, polynucleotide pharmaceutical composition, and, compound
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
JP7585233B2 (en) * 2019-04-30 2024-11-18 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Compositions Useful for the Treatment of Pompe Disease
ES3017234T3 (en) * 2019-09-25 2025-05-12 Lumosa Therapeutics Co Ltd Pharmaceutical composition comprising thrombolytic peptide-tetrahydroisoquinoline conjugate
MX2022005654A (en) 2019-11-20 2022-06-22 Chugai Pharmaceutical Co Ltd PREPARATION CONTAINING ANTIBODY.
EP4243855A4 (en) * 2020-11-16 2024-10-16 Avrobio, Inc. Compositions and methods for treating pompe disease
KR20230128462A (en) 2020-11-19 2023-09-05 제브라 덴마크 에이/에스 Process for preparing arimoclomol citrate and intermediates thereof
US20240318156A1 (en) * 2021-06-25 2024-09-26 Biomarin Pharmaceutical Inc. Compositions of beta-hexosaminidase variants and uses thereof
AU2023383441A1 (en) 2022-11-18 2025-06-05 Seismic Therapeutic, Inc. Fc fusion molecules and uses thereof
WO2024148276A1 (en) 2023-01-06 2024-07-11 Seismic Therapeutic, Inc. Protease variants and uses thereof
CN120118891B (en) * 2025-03-10 2025-11-21 广州青囊生物科技有限公司 Beta-glucosidase freeze-drying protective agent and application thereof in production of CK

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040161837A1 (en) * 1995-08-02 2004-08-19 Reuser Arnold J. Methods of purifying human acid alpha-glucosidase
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20080014188A1 (en) * 2004-02-06 2008-01-17 Zankel Todd C Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof

Family Cites Families (83)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4309776A (en) 1980-05-13 1982-01-12 Ramon Berguer Intravascular implantation device and method of using the same
US4749570A (en) 1981-12-31 1988-06-07 The Governors Of The University Of Alberta Targeting conjugates of albumin and therapeutic agents
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
DE3581528D1 (en) 1984-07-13 1991-02-28 Chiron Corp INSULINE-LIKE GROWTH FACTOR II.
CA1260858A (en) 1985-03-28 1989-09-26 Lawrence S. Cousens Expression using fused genes providing for protein product
US4902505A (en) 1986-07-30 1990-02-20 Alkermes Chimeric peptides for neuropeptide delivery through the blood-brain barrier
US4801575A (en) 1986-07-30 1989-01-31 The Regents Of The University Of California Chimeric peptides for neuropeptide delivery through the blood-brain barrier
US5470828A (en) 1987-12-24 1995-11-28 Gropep Pty. Ltd. Peptide analogs of insulin-like growth factor II
US5817623A (en) 1995-03-06 1998-10-06 Univ Colorado State Res Found Method for treating diabetic peripheral neuropathy with IGF-I or IGF-II
US5236838A (en) 1988-12-23 1993-08-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5549892A (en) 1988-12-23 1996-08-27 Genzyme Corporation Enhanced in vivo uptake of glucocerebrosidase
US6451600B1 (en) 1989-12-22 2002-09-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5977307A (en) 1989-09-07 1999-11-02 Alkermes, Inc. Transferrin receptor specific ligand-neuropharmaceutical agent fusion proteins
US6610299B1 (en) 1989-10-19 2003-08-26 Aventis Pharma Deutschland Gmbh Glycosyl-etoposide prodrugs, a process for preparation thereof and the use thereof in combination with functionalized tumor-specific enzyme conjugates
US5248606A (en) 1990-06-11 1993-09-28 Dowelanco Dna encoding inactive precursor and active forms of maize ribosome inactivating protein
US5401650A (en) 1990-10-24 1995-03-28 The Mount Sinai School Of Medicine Of The City University Of New York Cloning and expression of biologically active α-galactosidase A
US5356804A (en) 1990-10-24 1994-10-18 Mount Sinai School Of Medicine Of The City Of New York Cloning and expression of biologically active human α-galactosidase A
US5633235A (en) 1991-04-19 1997-05-27 Regents Of The University Of Michigan Triciribine and analogs as antiviral drugs
FR2676058B1 (en) 1991-04-30 1994-02-25 Hoechst Lab GLYCOSYLATED PRODUCTS, THEIR PREPARATION PROCESS AND THEIR USE IN THE TREATMENT OF CANCERS.
AU2368892A (en) 1991-07-29 1993-03-02 British Bio-Technology Limited Igf-ii analogues
US6270989B1 (en) 1991-11-05 2001-08-07 Transkaryotic Therapies, Inc. Protein production and delivery
US5258453A (en) 1992-01-21 1993-11-02 University Of Utah Drug delivery system for the simultaneous delivery of drugs activatable by enzymes and light
EP0646178A1 (en) 1992-06-04 1995-04-05 The Regents Of The University Of California expression cassette with regularoty regions functional in the mammmlian host
US5981194A (en) 1992-07-10 1999-11-09 University Of British Columbia Use of p97 and iron binding proteins as diagnostic and therapeutic agents
US5476779A (en) 1992-09-30 1995-12-19 University Of Maryland At College Park DNA encoding insulin-like growth factor II isolated from rainbow trout
EP0599303A3 (en) 1992-11-27 1998-07-29 Takeda Chemical Industries, Ltd. Peptide conjugate
US5633234A (en) 1993-01-22 1997-05-27 The Johns Hopkins University Lysosomal targeting of immunogens
JP3946246B2 (en) * 1993-02-02 2007-07-18 ゾーマ・コーポレイション Pharmaceutical composition containing bactericidal / permeability improving protein (BPI) and surfactant
US5798366A (en) 1993-05-13 1998-08-25 Monsanto Company Method for treatment of CNS-involved lysosomal storage diseases
US5704910A (en) 1995-06-05 1998-01-06 Nephros Therapeutics, Inc. Implantable device and use therefor
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5817789A (en) 1995-06-06 1998-10-06 Transkaryotic Therapies, Inc. Chimeric proteins for use in transport of a selected substance into cells
US6118045A (en) 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
US20020013953A1 (en) 1995-08-02 2002-01-31 Reuser Arnold J. Compositions and methods for treating enzyme deficiency
DK0852491T3 (en) 1995-09-29 2004-12-20 Biomarin Pharm Inc Use of heparinases to decrease inflammatory responses
US6348194B1 (en) 1995-11-13 2002-02-19 Ixsys Incorporated Tumor specific internalizing antigens and methods for targeting therapeutic agents
US6344436B1 (en) 1996-01-08 2002-02-05 Baylor College Of Medicine Lipophilic peptides for macromolecule delivery
WO1997026277A2 (en) 1996-01-22 1997-07-24 Creative Biomolecules, Inc. Morphogen analogs and methods for producing them
US6458574B1 (en) 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
US6020144A (en) 1996-09-12 2000-02-01 Symbiontics, Inc. Sustained delivery device comprising a Leishmania protozoa and methods of making and using the same
US6083725A (en) 1996-09-13 2000-07-04 Transkaryotic Therapies, Inc. Tranfected human cells expressing human α-galactosidase A protein
US6235874B1 (en) 1997-01-10 2001-05-22 Academia Sinica Production of biologically active recombinant insulin-like growth factor II polypeptides
US6329501B1 (en) 1997-05-29 2001-12-11 Auburn University Methods and compositions for targeting compounds to muscle
ATE359561T1 (en) 1997-06-02 2007-05-15 Univ Johns Hopkins COMPUTER METHODS USING FREE ENERGY CALCULATION FOR LIGAND DESIGN AND PREDICTION OF BINDING TARGETS
US6472140B1 (en) 1997-09-05 2002-10-29 The General Hospital Corporation α-2- macroglobulin therapies and drug screening methods for Alzheimer's disease.
IL135578A0 (en) 1997-10-29 2001-05-20 Genzyme Corp Compositions and methods for treating lysosomal storage disease
IL141312A0 (en) 1998-08-11 2002-03-10 Biosource Tech Inc Method for recovering proteins from the interstitial fluid of plant tissues
ES2312222T3 (en) 1998-12-07 2009-02-16 Genzyme Corporation POMPE DISEASE TREATMENT.
US6596500B1 (en) 1998-12-08 2003-07-22 The General Hospital Corporation Binding of retinoids to M6P/IGF-II receptor
US6638727B1 (en) 1999-01-26 2003-10-28 Cytyc Health Corporation Methods for identifying treating or monitoring asymptomatic patients for risk reduction or therapeutic treatment of breast cancer
US6770468B1 (en) 1999-09-14 2004-08-03 Genzyme Glycobiology Research Institute, Inc. Phosphodiester-α-GlcNAcase of the lysosomal targeting pathway
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
US6569661B1 (en) 1999-11-12 2003-05-27 Biomarin Pharmaceutical Inc. Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US6426208B1 (en) 1999-11-12 2002-07-30 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US6585971B1 (en) 1999-11-12 2003-07-01 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof
US20020081654A1 (en) 2000-04-07 2002-06-27 Sandrin Mauro Sergio Targeting hydrolase enzymes
EP2767291B1 (en) * 2000-07-18 2016-09-14 Duke University Treatment of glycogen storage disease type II
US20020142299A1 (en) 2001-01-09 2002-10-03 Davidson Beverly L. PTD-modified proteins
US7723296B2 (en) * 2001-01-18 2010-05-25 Genzyme Corporation Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
US20030004236A1 (en) 2001-04-20 2003-01-02 Meade Thomas J. Magnetic resonance imaging agents for detection and delivery of therapeutic agents and detection of physiological substances
WO2002087510A2 (en) 2001-04-30 2002-11-07 Symbiontics, Inc. Subcellular targeting of therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20040005309A1 (en) 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
AU2002347910A1 (en) 2001-10-16 2003-04-28 Symbiontics Inc. Methods and compositions for targeting proteins across the blood brain barrier
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US6905856B2 (en) 2001-12-21 2005-06-14 Genzyme Glycobiology Research Institute, Inc. Soluble GlcNAc phosphotransferase
US6800472B2 (en) 2001-12-21 2004-10-05 Genzyme Glycobiology Research Institute, Inc. Expression of lysosomal hydrolase in cells expressing pro-N-acetylglucosamine-1-phosphodiester α-N-acetyl glucosimanidase
WO2003086452A2 (en) 2002-04-05 2003-10-23 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy
US7485314B2 (en) 2002-05-06 2009-02-03 Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center Induction of antigen specific immunologic tolerance
EP1514106A4 (en) 2002-05-29 2007-05-09 Zystor Therapeutics Inc TARGETED THERAPEUTIC PROTEINS
US20050142141A1 (en) 2002-11-27 2005-06-30 Pardridge William M. Delivery of enzymes to the brain
ES2371913T3 (en) 2003-01-22 2012-01-11 Duke University IMPROVED CONSTRUCTS TO EXPRESS LISOSOMAL POLYPEPTIDES.
US20050026823A1 (en) 2003-06-20 2005-02-03 Biomarin Pharmaceutical Inc. Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US7842661B2 (en) 2003-11-24 2010-11-30 Novo Nordisk A/S Glycopegylated erythropoietin formulations
CA2553955C (en) 2004-02-10 2012-08-28 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
WO2005077333A2 (en) 2004-02-10 2005-08-25 University Of Florida Research Foundation, Inc. Gel-based delivery of recombinant adeno-associated virus vectors
US8017151B2 (en) 2004-09-07 2011-09-13 Board Of Regents Of The University Of Nebraska By And Behalf Of The University Of Nebraska Medical Center Amphiphilic polymer-protein conjugates and methods of use thereof
JP2009529885A (en) 2006-03-17 2009-08-27 バイオマリン ファーマシューティカル インコーポレイテッド Assays for detection of antibodies against lysosomal enzymes
CA2680189A1 (en) 2007-03-06 2008-09-12 Saint Louis University Modified enzyme and treatment method
JP5627571B2 (en) * 2008-05-07 2014-11-19 バイオマリン ファーマシューティカル インコーポレイテッド Lysosomal targeting peptides and uses thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040161837A1 (en) * 1995-08-02 2004-08-19 Reuser Arnold J. Methods of purifying human acid alpha-glucosidase
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20080014188A1 (en) * 2004-02-06 2008-01-17 Zankel Todd C Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
PASTORES G M ET AL: "Enzyme-replacement therapy for Pompe disease", PEDIATRIC HEALTH, FUTURE MEDICINE LTD, vol. 3, no. 1, 1 January 2009 (2009-01-01), pages 41 - 49, XP009167802, ISSN: 1745-5111 *
See also references of WO2010148253A2 *

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EP3075386A1 (en) 2016-10-05
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US20120148556A1 (en) 2012-06-14
EP2475376A2 (en) 2012-07-18
ES2758827T3 (en) 2020-05-06
WO2010148253A2 (en) 2010-12-23
ES2569514T3 (en) 2016-05-11
WO2010148253A3 (en) 2011-05-26
EP2475376B1 (en) 2016-03-30
EP3075386B1 (en) 2019-10-16

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